LifeMax Laboratories Inc a subsidiary of LifeMax Healthcare International Corporation, recently announced that a Novartis licensed investigational therapy, LM-030, has been granted pediatric disease designation by the FDA. The therapy is now ready to enter critical clinical trials for the treatment of Netherton Syndrome, cite sources.

LifeMax Laboratories is a clinical stage biotech company focused on treating rare diseases with limited or no therapeutic options. 

For the record, Netherton Syndrome is an autosomal recessive disease with symptoms including bamboo hair congenital erythroderma & abnormality in the immune system. It can be lethal in pediatrics due to weakened skin barrier that leads to severe, hypothermia dehydration, sepsis, hypernatremia and gross weight loss.

Most patients with Netherton Syndrome also suffer with immune system related disorders such as asthma and food allergies. As a result of persistent cutaneous infection, chronic erythroderma, metabolic disorders & malnutrition, failure to thrive is common in childhood. Over time the acuteness of the skin abnormality can fluctuate in older patients.

Larry Hsu, Co-founder and CEO, LifeMax, was reportedly quoted saying that the company is elated on receiving rare pediatric disease designation for investigational LM-030, which has demonstrated safety & clinical efficacy in a Phase I as well as phase II study. He added that receiving the rare pediatric disease designation is a significant step in the development of LM-030 for treatment of Netherton Syndrome, which is a severe genetic disorder with neonate onset.

As per sources close to the matter, rare pediatric disease is defined by the Food and Drug Administration as a disease affecting less than 200,000 people in the United States and the most severe, life threatening indicator in pediatric patients.

If NDA new drug application for LM-030 is approved, the firm may be eligible for receiving a rare pediatric disease priority review voucher. The voucher can then be redeemed to obtain priority review for any subsequent BLA Biological License Application or NDA and can be sold or transferred.

Source credits: https://www.lifemaxhealthcare.com/july-18-2019